Fernando Goldzstein Transcript — Nov. 18, 2025
Rachel Jones/NPF (00:01):
For this next session. The term diagnostic odyssey in the rare disease realm refers to the long, difficult and often years long process it takes to get a correct diagnosis. And then there’s the even more excruciating path to an effective treatment. When Brazilian businessman, Fernando Goldzstein’s son Frederico, was diagnosed with a rare brain tumor at age nine. His own journey towards an effective treatment led to a collaboration with Children’s National Hospital in Washington DC that seeks to speed up the research that can. It also yielded the launch of the Medulloblastoma Initiative, an international organization that seeks cure for these rare cancers by supporting life-changing scientific research. You can read Fernando’s full bio on our website at nationalpress.org. Fernando, thank you so much for joining us today.
Fernando Goldzstein/Medulloblastoma Initiative (01:12):
Thank you, Ray Rachel, very much. Thank you. The National Press Foundation for the opportunity to be here at this rare disease reporting fellowship. It’s an honor to be here. Good afternoon for who is in DC or Brazil? And for the others you
Rachel Jones/NPF (01:37):
Think in Malaysia and Kenya and all over the world.
Fernando Goldzstein/Medulloblastoma Initiative (01:42):
Yes, yes. So it’s my pleasure to be here and to share my story with you. I’m just going to share my screen here. Just a second. Can you all see?
Rachel Jones/NPF (02:03):
Yes.
Fernando Goldzstein/Medulloblastoma Initiative (02:05):
Okay. So I’d like to start with proposing a reflection. I know there are many people who is young here, but I would like to you think about what do you have most precious in your life? So for me and for who is a mother or a father, I think everybody will think about their kids. This is the most pressure thing we have in life. These are my kids, Frederico, Enrique, this is an old picture, but here they are. In 2015, my oldest son who is here in the picture was diagnosed with Mesoblast is a rare disease because cancer in kids, thanks God is rare. Although it happens. It happens. And Mesoblast stoma is the most common type of cancer in kids. It happens to 15,000 kids in the world every year. On that November 7th, 2015, we just completed 10 years from this date, we received this news that our son was diagnosed with this brain tumor, and at that time, our lives frozen. So the time stop for us, I mean, it’s very difficult to put in words what a father or a mother feel in the circumstances like this.
(04:09):
Then I started in this world of brain cancer and I realized it that kids with brain tumors were left behind by the society because believe it or not, the treatment is very old. It’s is from the eighties. So when this treatment that it’s being still used in all over the world, even in rich countries, it’s not a matter of richness. I mean, this is the only treatment available. This treatment was created in the eighties by the time we were using this kind of technology. So you can imagine how things evolve from that time, but not for kids with brain tumors. So the treatment is very toxic and ineffective because 30%, more or less of the kids relapse. And if they relapse, there’s no cure. And you might think that, well, we have 70% of the kids cured. Yes we do. However, the treatments are very, very toxic and the kids suffer from collateral effects that affect their quality of life, like they don’t grow or they have cognitive problems of even secondary tumors. So I realized that as I mentioned in the beginning of this slide when I was talking about this slide, that the kids with brain tumors were left behind. This is Frederico being treated. This is 2016 or 2017. He was nine years old when he was diagnosed. And this is exactly the average age of the kids who has me neuroblastoma, nine years old.
(06:12):
However, we three years after, actually he was being treated here and we taught and we were praying to be in the right side of the statistics. So although he might have collateral effects, of course we went to save his life. So we were hoping to be in the right side of the tracks. However, three years later, we realized that we weren’t in the right side of the tracks. So his tumor relapse, and as I mentioned in the beginning, if this tumor relapsed, there’s no cure. A hundred percent of the kids are going to die. We received this news in Boston because Federico was treated in Boston, although we live in Brazil, we treated him in 2015 in Boston. Then we went back to Brazil, live in Brazil, and sometimes we went to Boston for follow-ups. And in one of these follow-ups, the relapse was diagnostic. So the doctor said that there was nothing to do to save his life. They recommend that we go back to Brazil and just wait because was, as I mentioned, there was nothing to do.
(07:45):
However, giving, oops, sorry, up on Federico wasn’t an option for me. So I started to talking to a lot of people all around the world to try to see if there is something that could be done. Remember you that I was in Boston, which is one of the best medical centers in the planet. So having their opinion that there was nothing to do, it was, I mean you, I mean I remember thinking back then where I’m going to run to because I am already in one of the best places in the world, but I start talking to many people and I ended up reaching this doctor. His name is daughter Roger Packer. He is from the Children’s National Hospital in Washington DC as Rachel mentioned it before. So I talked to Dr. Roger Packer. He recommend us to do two clinical trials, one in New York, one in Washington.
(09:03):
So I dunno if probably you are familiar clinical trials, they are testing new treatments. So there is no scientific proof that things are going to work or not. So you are in unchartered waters, but you don’t have, and even it might be toxic, you don’t know, but you don’t have options. So you have to do something or the rise that the tumor is going to move ahead until the patient dies. So we did these two trials, but the problem was that the trials that we are doing, they weren’t specific for mesoblast stoma. There were kids with different brain tumors that was being accepted in this trial. So there was no trial specific for frederico’s disease. So I know of course I was hoping for the best, but I had to be prepared for the worst. So on the back of my head, I knew that I should do something else.
(10:20):
Then was the time that I decided actually before that, sorry about that. I start talking with Dr. Parker about what could be done. Dr. Parker, what can we do if I make a donation? Can we kind of speed research? Can we do something to have a treatment for my son in of course in time to save his life? Because we know that kids with relapsed neuroblastoma, they will last 2, 3, 4 years at the most. So I made a donation. I gave him X amount of money and I was very impressed because he start working with two or three other institutions. So Frederic was under these two clinical trials that I mentioned, but I wanted to be prepared for something, right? If something bad happens and then he start working with these other institutions, I was very impressed and I decided to do something else. And then when I decided to create the BLA initiative, and I’m going to show a two minute video for you now that will give you a better sense about what middle blastoma initiative is. So let make sure that we have sound in this video. Can you hear I 30% of the kids will have metal blastoma. They don’t survive and there’s no cure yet. So we decided to do initiative that we call blato initiative to change that
(12:22):
You cannot hear In 2015, my son got can hear and it’s very, very tough to see the kids surfing. He is a very smart guy. He likes to travel very much. He likes to read, he loves the school as well. Fred has been a warrior and I decided that fighting the pediatric brain tumor will be the goal of my life from now.
Speaker 3 (13:05):
Philanthropy is so critical to us. We wouldn’t be able to move in many different ways. We wouldn’t be able to move those innovatively and we wouldn’t be able to move as quickly. And the longer we delay, the more children get hurt by the risk of losing their lives or the qualities of lives because we don’t have answers for the,
Speaker 4 (13:26):
In 2024, we launched two FDA approved trials of new treatments, a cell therapy and an mRNA vaccine. Doctors Mitchell and Seor will lead the trials for children with relapsed brain tumors, both hold the potential to transform the field and save lives. Neither would’ve happened without the MBI in its donor support.
Fernando Goldzstein/Medulloblastoma Initiative (13:49):
There is a quote in the TUD that says, wherever saves the life, saves the entire world.
Rachel Jones/NPF (14:14):
Thank you for that.
Fernando Goldzstein/Medulloblastoma Initiative (14:16):
Thank you. So as we mentioned in the video, we were able to approve two clinical trials in two years and a half, which is a record speed normally takes seven to 10 years. So one clinical trial, this one is already not only approved by the FDA in two years and a half, but it’s already treating kids with relapsed mesoblast stoma. So this is in specific treatment for relapsed mesoblast stoma and kids are already being treated. So kids that did not have any other option. So this is something that we created from scratch. This is the second trial. This is already approved by the F fda, but it’s not open. It’s not recruiting yet. And then this is just the first trial. This is a document from NIH that our trial is being recruiting kids. Then we have a third trial on our pipeline and the fourth trial on our pipeline.
(15:25):
And not going to go into scientific details because I don’t think it’s the case. And I have to say that I’m not doctor and I’m not scientist, right? I’m just a father trying to save my son and thousand of other kids. These are institutions that belongs to under our umbrella. So as I mentioned, we start with two, three, now it’s 16 labs in 12 institutions and all over the us, Canada and one institution in Germany. So MBII, I’m going to, oops, what I did here, sorry. Okay, when I started, when we started this, we didn’t know exactly where we are going to be in two years, two years, four years. I would never imagine that MBI would be a global movement.
(16:39):
And also I have been going to the US many times and we realized that we created something very different. Something that it’s not, I wouldn’t say unique, but there might be other. But it’s very, very different because these labs, these 16 labs, they have to follow some rules, for example, to be funded by us. First of all, everyone has, its part of the puzzle, right? That’s why we have a puzzle here. So we are going to solve this treatment and we are going to solve this disease if we work together. This is our philosophy. So we have to work together and we have to work fast so everyone has it part of the puzzle. Also, it’s all about teamwork, right? There is no silos, there is no duplicate work. So everyone has to share its information on. So that’s another thing that is very important in our model.
(17:55):
Another thing is the sense of urgency. For example, you might have heard about other initiatives like MBI that want to find a cure of other disease, but most of them they are created as a tribute in memorial for something who already passed away. MBI was created to save Fred Rico and of course now thousands of other kids. But the sense of urgency if you are trying to do something to save one specific patient is completely different if you are doing something in memorial of a patient. So we have a kind of sense of urgency, which is very different of course, you know that we cannot speed science. The cells are, they have a time to duplicate. You cannot speed that, but you can cut the red tape, you can speed the bureaucracy. So you can do this as a teamwork. Many things are going, doing together instead of doing a sequence.
(19:06):
So this is kind of the magic of our model. And another thing that I have to highlight is that most of the initiatives that I mentioned that they do as a rebut or in memorial, they give what they call scattered grants. They have a scientific committee and they accept or don’t accept grant requests from everywhere. We don’t give scattered grants because we think that if we give one grant here and one grant there, at the end of the day we don’t consolidate and we don’t have effectiveness. So we only fund this group of labs. If there is a new lab that wants to join us and we think that they’re going to add for our work, okay, we will have a new lab, but this lab has to comply with the rules of sharing formation, no silos, blah blah blah, blah, blah, blah. So this is a new model, a new approach. And I think that’s the main reason we have been able to succeed in a very short time. So here
(20:31):
This is a very important article that was published in January of this year, MIT, which is of course a very important institution. They are recognizing us in this article that MBI might be a playbook not only to of course, not only to find the cure of neoblastoma, but many other diseases. And I’m going to share you another clip very quickly that are going to give you an idea about the awareness that MBI is having in the last few months. So I’m going to share it to you. Another clip and then I’m almost finishing. Can you hear?
Rachel Jones/NPF (21:28):
Not yet.
Speaker 5 (21:31):
The Mell stomach initiative, it wasn’t difficult for me and my family. Make the decision of trying.
Speaker 3 (21:43):
If you don’t have a startup and you’re not going to be able to start things that a novel they need and you see kids diet, this disease and brain tumors, this isn’t very, this is common sense, something different and you got to do it differently or we shouldn’t be in the business.
Speaker 6 (22:06):
Something that can go from zeros control the majority of patients alive within that two year period. That was the goal and that was the funding and that was the development of something called the Medulloblastoma initiative, which is
Fernando Goldzstein/Medulloblastoma Initiative (23:00):
MBI. It is an initiative that I created. By the way, I’m not doctor or scientist, I am a father. So my son was diagnosed with met neuroblastoma 10 years ago. Believe it or not, the treatment for neuroblastoma and other pediatric brain cancers are from the eighties. And we were able to approve two clinical trials already. So even though we have three years old, only, we approved it two clinical trials and we are treating kids already
Speaker 7 (23:33):
Tonight we share the story of one father who’s creating a global network of scientists and researchers while raising millions to fast track a cure for his son and kids around the world. It was 10 years ago, more or less I learned that kids with brain tumor were left behind. Less than 4% of the US Federal cancer research is for pediatric research. It’s a model
Fernando Goldzstein/Medulloblastoma Initiative (23:59):
That brings together the best minds in the field. MBI is all about collaboration and synergy. So the scientists to be funded by us, they have to work as
Speaker 7 (24:11):
A group. You can learn more or donate to the Medulloblastoma initiative by visiting MB initiative.org.
Fernando Goldzstein/Medulloblastoma Initiative (25:19):
Well, to finish, I’d like to just give a glance about Frederico. He is fine. He is 19 years old now. As you can see, he didn’t grow much, but he has all the functions and he does not have cognitive problems. So he is a warrior. And actually, thanks God, he is an outlier because he is living with this relapse for many years, which is, he already had two relapse after that. But it’s not common to see kids surviving that much. But thanks God, he is doing alright. And I will finishing telling you that was a great pleasure to be here and I hope you understand about our model and I hope if you have the opportunity to spread the word in your countries about us or you want to get in touch to have more details. So thank you very much. Thank you, Rachel. Very much
Rachel Jones/NPF (26:33):
Nando. This is such an extraordinary example of what I have been sharing with the journalist over the past day, and that is in my experience as a journalist, the commitment and focus and dedication of parents and caregivers in the rare disease space has just been extraordinary for me to observe. I told the other day that I never saw more hugs given in any conference setting than I saw at the Nord conference. People are so supportive of each other and so determined to help each other. So I want to save as much time as I can for questions. And I see we have some good ones already. So I’m going to ask first Simon Spichak to ask his question and then followed by Alicia Gar. So Simon, go ahead please.
Simon Spichak | Being Patient (27:30):
Yeah, I’m just curious, in terms of setting up clinical trials for children, is it more complicated than running trials for adults, especially when you have that added urgency and is there something surprising or unexpected you learned about the process?
Fernando Goldzstein/Medulloblastoma Initiative (27:47):
Thank you for the question, Simon. Actually, yes, it’s more difficult, but the way we do, I don’t participate directly in the scientific process, although I am following up everything that happens, but all the details of the process and everything, what I do for MB, I do the awareness, I do the fundraising, and Dr. Parker is in charge of leading all these labs that you see and all this process. So I cannot, unfortunately, I cannot go into the details about the setbacks that they have. I know with kids it’s always more delicate, it’s always more concerning, but it shouldn’t be because for some diseases what I learn, I’m going to be maybe a little bit hard in the way of, I’m going to tell, but if the kids is going to die anyway, why you should not take some risks. So what I heard from the field, it should be more, it should be faster. Because if you have situations that you don’t have option, I think the parents would like to take the chances and have a trial sooner than later, which will be too late. But this is not an official opinion about MBI. I’m just talking with friends here. So I think Dr. Packer should give an official opinion about that and I’ll be happy to ask him to answer any questions to Rachel.
Rachel Jones/NPF (29:50):
Thank you. Thank you, Alicia.
Alicia Garceau | Independent (29:53):
Hi. Thank you so much for being here. I just had a question about, we’ve heard so much about cancer research being impacted by the cuts to NIH, and I’m wondering if any of your trials have been impacted, particularly the ones with the mRNA vaccine?
Fernando Goldzstein/Medulloblastoma Initiative (30:13):
Yeah, it seems, again, I don’t have the details. I know that it might have some impact. I don’t know if th already have impact, but that, for example, that interview that I gave to M Ns, sorry, Ms. NBC, they, I don’t know if you paid attention, but they were talking about, they wanted to know about the mRNA because the cuts just happened just one or two weeks before that interview. And my opinion on that interview was that, of course I’m totally against that Kurtz because, and first of all, when you talk about vaccine, sometimes people get confusing and they think that there’s going to be a vaccine to a herd to, for all the population, this word vaccine, it’s a little bit confusing, at least in Brazil, but I think in the US too. So the vaccine, the RNA vaccine is something that is going to be given for people with cancer, right?
(31:30):
For cancer does not come back or even to treat the cancer. So that’s going to be an option that we are going to have clinical trials, and that might be the cure of my son and many other and kids. And it’s an approach different from chemotherapy, which is chemotherapy. You kill the bad cells, but you killed the good cells, right? And it’s one size fits all. So where a vaccine is something tailored for each patient. So of course it shouldn’t be cut it. But to answer your question, I don’t know yet if we are going to be affected or not our trial because we don’t have federal funds in our trial. But the doctors that are funded by us, they have other trials that are funding from federal funds. So they might be affected, but I don’t know yet.
Rachel Jones/NPF (32:37):
I’m going to ask Nokukhanya to speak next. And please introduce yourself to Fernando, are you there? No, Kuan?
Nokukhanya Musi – AimienohoInhlase | Centre of Investigative Journalism (32:52):
Yes I am. Hello Fernando. And hello to the rest of the fellows. And hi, Rachel. I think I’m more interested in finding out the emotional, mental toll the whole experience had on not just you, but the entire family. How did you cope during the entire, I mean, from the moment your son was diagnosed and they’re running around from Boston to Brazil, how was that on your mental health?
Fernando Goldzstein/Medulloblastoma Initiative (33:39):
Okay, thank for the question. No, yeah. I mean when somebody approached me and listen the story and maybe and the person say, oh, I can’t imagine what you are feeling. I always answer that. No, you cannot imagine. So it’s impossible to imagine. I mean, it’s a nightmare that does not stop. So you live all your days and all your hours with this, with this, how can I say? With this
Nokukhanya Musi – AimienohoInhlase | Centre of Investigative Journalism (34:17):
In suspense?
Fernando Goldzstein/Medulloblastoma Initiative (34:18):
Yeah, in suspense, yeah. But I decided to fight. I decided don’t accept to not accept. And I think that, and my son is very brave as well. For example, he did already for I think four had surgeries. And even having, now he’s 19, but he’d had 9, 12, 14, and he was very brave. So he never say, no, I don’t want to go. He never said that. He always went to the operating room. Very brave in a very brave way. And that gives me lots of strength. And also by him seeing my fight to find the cure of his disease, probably I’m giving him strength as well. So I could be just stay desperate or stay in Brazil and Frederico wouldn’t be alive anymore for sure. And I think what keeps me with my hand, with my head on my shoulders is that is this fight, right? Knowing that we have new trials and we are going to have more trials. I mean, it’s not very common what I’m doing and the results we’re having, but I mean, that’s the way I found to try, how can I say, to face this terrible situation. But of course it’s very hard. It’s very difficult.
Nokukhanya Musi – AimienohoInhlase | Centre of Investigative Journalism (36:04):
Incredibly brave of you. Thank you.
Fernando Goldzstein/Medulloblastoma Initiative (36:07):
Thank you very much.
Rachel Jones/NPF (36:08):
I want to jump in quickly to ask you to expand a bit on something you said. You said, I’m not a scientist, I’m not a researcher, I’m just a father. But what we have learned from hearing from other people is that parents have to almost become researchers. They have to educate themselves, the parents of baby KJ in Philadelphia or in Pennsylvania. They had whiteboards up on their walls and they’re making notes to themselves. So please give us some insight into the process you took to sort of understand what was going on with Federico.
Fernando Goldzstein/Medulloblastoma Initiative (36:54):
Yeah, yeah, that’s a very good question. And I think I never was asked this question, and it’s very interesting. I understand that I am in touch with more than a hundred families with parents, with kids with relapsed blas stoma for all over the world. So I can see many kinds of parents, desperate parents, parents who knows a lot. And so everyone has its own reaction, but I understand what you mean. But with me, it happens something a little bit different, which might be for everyone has its own way to react. But when he was diagnosed with this disease, then we have a protocol, then you don’t have many things to choose. You have to follow the protocol. You are not going to create something if you have a protocol that saves 70 to 75% of the kids. So you go through the protocol, I went to the US now in Brazil, I went to the US because there is a kind of radiation that is less toxic, but we don’t have it in Brazil, we don’t have it.
(38:24):
We just have in rich countries, I would say, which is less toxic. So that’s why I ended up in the us. But I was following a protocol When the tumor relapse, then you have a problem. Because I didn’t reach to Dr. Parker on the first two weeks, and I was talking to many other, many other doctors. And then when you are in Unscattered, others, when you don’t have a protocol, then the doctors tell you different things. And that is very difficult. And one doctor say, you can’t do the treatment X, another doctor say, why don’t you do the treatment Y and then you need to. But even if I start studying, I would never know enough to take such a decision. Never, never. Of course, I spent my nights in the hotel in Boston studying, studying. So my best friend there was the lady that did the cleaning of the lobby at three o’clock in the morning. So I talked to her every night because I was in the business center studying what happens with kids, with the kid with relapsed metal stone. But it’s impossible for me to know enough to decide.
(39:57):
I think when I met Dr. Packer and I decided to trust him, that was my best decision because I think at the end of the day, you have to trust somebody, somebody who is an expert, somebody who has connection, somebody who knows everything that it’s happening. Not in the medical Congress, not in the medical events, but Dr. Parker knows what’s happening before the articles are published, right? So when I met Dr. Parker and decided to trust him, then I say, okay, the way, I’m just going to do the awareness and I’m going to do the fundraising to support you, to support your work. So it’s a different, I wouldn’t say that I stopped studying about neuroblastoma, but I decided to trust him.
Rachel Jones/NPF (41:04):
What a powerful image of you and the cleaning lady leaning on each other. You leaning on her to have someone to talk to about this? I’m going to ask Stephen Kenechi to ask his question. Introduce yourself and ask your question.
Stephen Kenechi | TheCable (41:26):
Hi, my name is Stephen Kenechi from Nigeria. It’s a very, very powerful story. You’ve shared Fernan. I really recommend your relentlessness in going about the pursuits of research for your son, your son’s condition. Now, you spoke about some of the bism and work, you talked about achieving two clinical trials in 2.5 years. You talked about 16 labs in 12 institutions as beneficiaries of the M Bism funding. I need you to shed some light on how you went about fundraising. You mentioned that much of the activities are around fundraising is something you achieve by yourself after creating the nonprofit. How did you go about mobilizing the resources to supercharge NBIS work within such a short timeframe? And what lessons do you think resource limited and rare disease communities can draw from it, especially in the global south?
Fernando Goldzstein/Medulloblastoma Initiative (42:36):
It was hard for me to understand you, Stephen, but you are talking about the fundraising, how I could have the resources to do, right?
Stephen Kenechi | TheCable (42:47):
Yes, absolutely. What are the things you think your story, your journey holds for resource limited communities in the global south dealing with red disease?
Fernando Goldzstein/Medulloblastoma Initiative (43:01):
Yeah, you are talking about global salt, but I just have to remember you that our research is not in the global salt. Our research is in the us, Canada, and Germany. So I start with the donation, which wasn’t a big donation, not enough to do of course, what we were doing. I raised much, much more money than that. So I don’t know exactly. I don’t know exactly how can I answer? Because for example, people ask what, there is no lab from Brazil. I am from Brazil, I am in Brazil right now, and we don’t have labs from Brazil. Why? Because the labs are not equipped to do the kind of research we need to do. So we raised it so far, $12 million MB, I raised it so far, $12 million. But we didn’t expand money in building a lab because if you wanted to do this in Brazil, we would have to spend maybe a very big chunk of this money building a lab in Brazil or building a lab in Nigeria.
(44:37):
And given MBI is something that I wanted to do something very fast and very effective, the best thing would be where are the labs? Where are the best labs? Where are the best minds? And let’s fund them. And sometimes people say, wow, but Brazil is not a rich country and you are sending money to the us. And I say, yes, I’m not funding the us. We are funding the cure of one disease that is going to benefit kids all over the world. It is just a matter of being more effective and fast. So if someone in Nigeria want to find a cure of a rare disease, maybe the best way would be to find where is the best doctor? Where is the best scientist in this disease? Probably he is not going to be in Nigeria or in Brazil, probably is going to be in the north because the things are like this. And then finds a way to try to raise money to fund this research. So it is exactly the way I said, how can I say? I challenged Dr. Parker. I said, Dr. Parker, I need the treatment in one year. Can you come with the treatment in one year? And he said, one year it’s impossible, maybe two years. And then I start to making very big movements in Brazil to try to fund and then I’m doing in the US as well. But yeah, I dunno if I answer your question. I don’t want to actually,
Rachel Jones/NPF (46:29):
I think that you did because what you’ve demonstrated is even if it’s a country or a region that does not have the infrastructure, does not have the scientists or whatever, making those collaborations, making those connections, supporting the research that is going on in Boston that may eventually help people in Nigeria and Brazil is equally important. I think that’s a pretty strong message. I’m going to introduce you to Marsha Uck simply because she says in the comment that you answered her question, but Marcia, go ahead and introduce yourself to Fernando.
Marcia Moyana | Freelance (47:18):
Hi. Hello, Mr. Fernando meet you all. Thank you for this fellowship. It’s a great time to learn things. And Mr. Fernando, you talked about something. It was almost my question for you. You are, well, it’s about the science and researching all over the world and the high science, because you mention it in your response now you about the labs, the high labs in Brazil, we don’t have hi labs and therefore you don’t found, you don’t put money here in Brazil. Well, I’m a journalist in Brazil and my interesting in this country, but you can please, I read my question here. How do you assess the overall state of science research in rare disease and the production of results for treatments and what is the state of research into rare disease in Brazil? And wouldn’t it be ing to invest a foundation in Brazil? I mean, the researchers don’t have effects structure to work in Brazil. They don’t have money, have the conditions. And so that’s my question.
Rachel Jones/NPF (49:37):
What she’s saying is, it’s an interesting point. It is. Collaborations are great, but is it possible to have an avenue where you’re supporting young scientists in Brazil? Yeah,
Fernando Goldzstein/Medulloblastoma Initiative (49:53):
Yeah. No, I can answer. Thank you for the question, Marcia. And yeah, I think one thing that is very important, and I’m going back to actually to Stephen question from Nigeria and ask Mars as well, but I think one thing that’s very important is focus. Focus, right? We live in a country with have lots of problems and kids, kids are misdiagnosed or maybe kids don’t have access to treatments. So that happens in Brazil, that happens in Nigeria, Nigeria. And that happens in many other undeveloped countries. And people say, but why? You don’t do something, why for example, why you like Mar’s? Question. Why don’t you do something for the young scientists? And my answer, it’s the following. We have to have focus. If we want to find the cure of mesoblast stoma, which nobody did yet, and which the last treatment was from the eighties, if you really want to do it, we have to focus on that.
(51:03):
Because if we try, okay, let’s do something for this other cause and that other cause, so you don’t have focus, you don’t get anywhere. So it seems to be a little bit hard, but that is the reality. The resources are limited and we have a mission. We have to find the cure of medulloblastoma and save these kids. But my purpose of life after we find the cure of Medulloblastoma, because it’s not a matter of if we find, but when we find then I’m going to devote my life to probably do something for, have more MRIs in Brazil, have better treatments for the kids in Brazil or in Nigeria or whatever. But now we have to focus, otherwise we are not going to find the cure of mesoblast stoma. So I think for example, and answer a little bit to Stephen question and Marc question, what we just did to try to help Brazil, we are doing some collaboration, how can I say collaboration?
(52:21):
I don’t know the word in English, but we have lots of knowledge because we have a constellation of scientists, the best in the world. So these scientists, we are having agreements with hospitals in Brazil, and these scientists are going to share their knowledge in webinars with Brazilian doctors in Brazilian hospitals. And then that we can do in Nigeria, we can do in other places. And because of course the money came from Brazil, the clinical trials when they start, thanks God, they are going to succeed in the US and we need to go to other countries. The first country we will go is to Brazil to try to give back to Brazil. But that’s why Marcia, we don’t focus in like, oh, let’s do something for a lab in Brazil. Let’s train the Brazil. That’s not our mission at this moment. And we have to focus. It’s hard, but is the right thing to do if you want to get there. Right.
Rachel Jones/NPF (53:33):
Amazing. Alex Ababio actually has an important question to ask. Alex, are you there? You may be having my problems. So let me ask it. How did your son’s rare disease impact his education and social life while growing up in Brazil, particularly in terms of school attendance, academic performance, peer relationships, et cetera? Alex’s from Ghana,
Fernando Goldzstein/Medulloblastoma Initiative (54:06):
This is your question, Rachel,
Rachel Jones/NPF (54:08):
It’s from Alex Ababio. He’s a reporter.
Fernando Goldzstein/Medulloblastoma Initiative (54:12):
Okay, I’m sorry, I’m not seeing the chat here. I’m sorry. Yeah, that’s a very good question. As I mentioned at the beginning, there are some kids who has cognitive effects. Frederico didn’t have, Frederico is very smart, but he was already before the diagnosis, before nine years old, he always was a quiet guy. So he, like in Brazil, every kid likes to play soccer, every kid, but Frederico didn’t. He preferred to read books. So he is kind of intellectual. And this disease of course didn’t help him because although he has no problems in school, but he has problems. And of course this is off the records because I don’t like to tell this for the press, I don’t like to expose him, so I ask you to keep between us. But he has of course, social issues, like very, very few friends. It’s very hard because he is short, he’s different. And he has been more than 50% of his life under treatment, under surgeries. So it’s very, very hard, very hard.
Rachel Jones/NPF (55:37):
Well, he is blessed and fortunate to have a father whose persistence, whose dedication and generosity in wanting to support other children as well is incredibly impressive. I want to ask you one last question, Fernando, while we have you. And that is, as you have viewed the coverage of not only MBI, but just rare disease in general, what are some stories that you want to see? What are some ways of covering these issues that you would like the journalists to consider as they move ahead?
Fernando Goldzstein/Medulloblastoma Initiative (56:25):
Wow, that’s a tough question. I think I can give the perspective of brain tumors, pediatric brain tumors, especially. I think people don’t realize yet that the treatments are old and less than 4% of the, this is before this administration. Now, I don’t know exactly how is how it is, but 4% of the money in the US for research, federal money goes to pediatrics, 4%, 96% goes to adults. And of course the cancers that the focus are in, breast cancer, lung cancer, prostate cancer, which are much more frequent, right in the population. But for example, if you have prostate cancer, that can be cured 95% or maybe 98, I don’t know exactly the numbers and the money goes to research for less medications with less collateral effects, which I think is wonderful. I don’t think it’s wrong, but I think we have kids with treatments from the eighties, right? From the eighties, which are very toxic. And so I think the society does not know that or don’t know enough. So I can’t, by this perspective, I would like that the press could communicate that more. Right.
Rachel Jones/NPF (58:06):
Well Fernando, you are a businessman. You say you’re not a scientist, but you are a father. You are a rare disease parent. And you have also today become an advisor for journalists around the world in speaking about the Medulloblastoma initiative and about the need to speed up the diagnostic process for children and families. So I thank you so much for joining us today.
Fernando Goldzstein/Medulloblastoma Initiative (58:39):
Thank you, Rachel. Thank you all for listening to me. It was a great pleasure and a great honor to be here with you today.
Rachel Jones/NPF (58:49):
And I will be in touch with you, Fernando. Take care.
